Evidence map›Paper›PMID 31917835›Full record

Trial reportThe Journal of clinical endocrinology and metabolism2020

Once-Weekly Somapacitan vs Daily GH in Children With GH Deficiency: Results From a Randomized Phase 2 Trial.

Lars Sävendahl, Tadej Battelino, Meryl Brod, Michael Højby Rasmussen, Reiko Horikawa, Rasmus Vestergaard Juul, Paul Saenger, REAL 3 study group

Erratum issued Registry-linked trialOpen access · hybridAbstract readClinical Trial, Phase IIComparative StudyMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The Journal of clinical endocrinology and metabolism, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT02616562 (A Randomised, Multinational, Active-controlled,), which is not on this map. Cited by 40 papers, 7 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
40citing papers in PubMed, 7 pooled it
4.8field-weighted citation impact, top 4% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02616562 phase2completednot on this map

A Randomised, Multinational, Active-controlled, (Open-labelled), Dose Finding, (Double-blinded), Parallel Group Trial Investigating Efficacy and Safety of Once-weekly NNC0195-0092 Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency

TypeinterventionalSponsorNovo Nordisk A/SRan2016 to 2024Enrolled76ConditionsGrowth Hormone Disorder, Growth Hormone Deficiency in ChildrenArmssomapacitan, Norditropin® FlexPro® pen
3 · Its place in the literature

Who cites it

40 citing papers in PubMed, 7 syntheses or guidelines pooled it, 59 citations in OpenAlex.

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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

8 authors at 6 institutions in 5 countries.

Lars SävendahlDepartment of Women's and Children's Health, Karolinska Institute and Pediatric Endocrinology Unit, Karolinska University Hospital, Stockholm, Sweden.
Tadej BattelinoUMC-University Children's Hospital, and Faculty of Medicine, University of Ljubljana, Ljubljana, Slovenia.
Meryl BrodThe Brod Group, Mill Valley, California, US.
Michael Højby RasmussenNovo Nordisk, Global Development, Søborg, Denmark.
Reiko HorikawaNational Center for Child Health and Development, Tokyo, Japan.
Rasmus Vestergaard JuulNovo Nordisk, Global Development, Søborg, Denmark.
Paul SaengerUniversity Hospital, Mineola, New York, US.
REAL 3 study group
University of Ljubljana · SIWinthrop-University Hospital · USKarolinska University Hospital · SENational Center For Child Health and Development · JPNovo Nordisk (Denmark) · DKBrod Group · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

contextDaily growth hormone (GH) injections can be burdensome for patients and carers. Somapacitan is a long-acting, reversible albumin-binding GH derivative in development for once-weekly administration in patients with growth hormone deficiency (GHD).

objectiveThe objective of this study is to evaluate the efficacy, safety, and tolerability of once-weekly somapacitan vs once-daily GH.

designREAL 3 is a multicenter, randomized, controlled, double-blind (somapacitan doses), phase 2 study with a 26-week main and 26-week extension phase (NCT02616562).

settingThis study took place at 29 sites in 11 countries. PATIENTS: Fifty-nine GH treatment-naive prepubertal children with GHD were randomly assigned; 58 completed the trial.

interventionsInterventions comprised 3 somapacitan doses (0.04 [n = 16], 0.08 [n = 15], or 0.16 mg/kg/wk [n = 14]) and daily GH (0.034 mg/kg/d [n = 14]), administered subcutaneously.

main outcome measuresThe primary end point was height velocity (HV) at week 26. Secondary efficacy end points included HV SD score (SDS) and insulin-like growth factor-I (IGF-I) SDS.

resultsAt week 26, mean (SD) annualized HV for the somapacitan groups was 8.0 (2.0), 10.9 (1.9), and 12.9 (3.5) cm/year, respectively, vs 11.4 (3.3) cm/year for daily GH; estimated treatment difference (somapacitan 0.16 mg/kg/week-daily GH): 1.7 [95% CI -0.2 to 3.6] cm/year. HV was sustained at week 52, and significantly greater with somapacitan 0.16 mg/kg/week vs daily GH. Mean (SD) change from baseline in HV SDS at week 52 was 4.72 (2.79), 6.14 (3.36), and 8.60 (3.15) for the somapacitan groups, respectively, vs 7.41 (4.08) for daily GH. Model-derived mean (SD) IGF-I SDS for the somapacitan groups was -1.62 (0.86), -1.09 (0.78), and 0.31 (1.06), respectively, vs -0.40 (1.50) observed for daily GH. Safety and tolerability were consistent with the profile of daily GH.

conclusionsIn children with GHD, once-weekly somapacitan 0.16 mg/kg/week provided the closest efficacy match with similar safety and tolerability to daily GH after 26 and 52 weeks of treatment. A short visual summary of our work is available (1).

Indexed as

BiomarkersChildChild, PreschoolDouble-Blind MethodDrug Administration ScheduleDwarfism, PituitaryFemaleFollow-Up StudiesHuman Growth HormoneHumansInsulin-Like Growth Factor Binding Protein 3Insulin-Like Growth Factor IMalePrognosisBiomarkersHuman Growth HormoneIGF1 protein, humanIGFBP3 protein, humanInsulin-Like Growth Factor Binding Protein 3Insulin-Like Growth Factor Igrowth hormonegrowth hormone deficiencygrowth hormone replacement therapylong-acting growth hormonesomapacitantreatment burden

Identifiers

PMID31917835
PMCPMC7069655
OpenAlexW2999234491

What OpenQuestion holds

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LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.